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在英国出生:一个旨在改善和传播有关华氏巨球蛋白血症知识的登记处。

Born in the UK: A registry to improve and broadcast knowledge on Waldenström macroglobulinaemia.

机构信息

Department of Hematology, University Hospital of Reims and UFR Médecine, Reims, France.

出版信息

Br J Haematol. 2023 Jun;201(5):809-810. doi: 10.1111/bjh.18695. Epub 2023 Feb 16.

Abstract

Registries constitute an interesting source of real-world data and bring complementary information to randomised controlled trials. They are of particular importance in rare diseases such as Waldenström macroglobulinaemia (WM), which can present with various clinical and biological features. In their paper Uppal and colleagues describe the development of the Rory Morrison Registry, the UK registry for WM and IgM-related disorders and highlight the profound changes in therapies both at first-line and relapsed settings in the recent years. Commentary on: Uppal E. et al. The WMUK Rory Morrison Registry for Waldenström Macroglobulinaemia: the growth of a national registry for a rare disorder. Br J Haematol. 2023;201:905-912.

摘要

登记处是真实世界数据的一个有趣来源,为随机对照试验提供了补充信息。它们在瓦尔登斯特伦巨球蛋白血症(WM)等罕见疾病中尤为重要,因为该病可能具有多种临床和生物学特征。在他们的论文中,Uppal 和同事们描述了 Rory Morrison 登记处的发展,该登记处是英国 WM 和 IgM 相关疾病的登记处,并强调了近年来一线治疗和复发治疗中治疗方法的深刻变化。评论:Uppal E.等人。WMUK Rory Morrison 登记处用于瓦尔登斯特伦巨球蛋白血症:罕见疾病国家登记处的发展。英国血液学杂志。2023;201:905-912.

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